Recruiting

FALCONSGT-212 Safety and Tolerability in Friedreich's Ataxia

What is being tested

SGT-212

+ SGT-212

Drug

Who is being recruted

Brain Diseases+20

+ Central Nervous System Diseases

+ Cerebellar Diseases

From 18 to 40 Years

+16 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1

Interventional

Study Start: October 2025

See protocol details

Summary

Principal SponsorSolid Biosciences Inc.

Study ContactSolid Biosciences Clinical TrialsMore contacts

Last updated: June 22, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: October 22, 2025

Actual date on which the first participant was enrolled.

This clinical trial is exploring a new treatment approach for people with Friedreich's ataxia (FA), a genetic condition that affects the nervous system and movement. The study is testing the safety and tolerability of a new drug called SGT-212. It is a first-of-its-kind study, meaning it is the initial time this treatment is being tested in humans. The trial aims to find the most appropriate dose that can be safely administered to participants. Understanding and improving how treatments can support those with FA is crucial, as this condition currently has limited treatment options. In this study, participants receive SGT-212 in two ways: directly into a specific part of the brain known as the intradentate nucleus and through an intravenous (IV) drip. The study will last about five years, during which the effects of the drug will be closely monitored. Researchers are particularly interested in how well participants tolerate the treatment and whether it causes any side effects. While the study focuses primarily on safety, it also provides an opportunity to gather early insights into how the treatment might help manage FA symptoms over time.

NCT07180355

Principal SponsorSolid Biosciences Inc.

Study ContactSolid Biosciences Clinical TrialsMore contacts

Last updated: June 22, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

10 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 40 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Brain DiseasesCentral Nervous System DiseasesCerebellar DiseasesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNutritional and Metabolic DiseasesPathologic ProcessesSigns and SymptomsSpinal Cord DiseasesSpinocerebellar DegenerationsPathological Conditions, Signs and SymptomsNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemDyskinesiasDisease AttributesMitochondrial DiseasesGenetic Diseases, InbornAtaxiaFriedreich AtaxiaMovement DisordersRare Diseases

Criteria

4 inclusion criteria required to participate

Has a clinical and genetic diagnosis of FA

Has a staging score of >= 1 but < 6 on the Friedreich's Ataxia Rating Scale (FARS) Functional Disability Staging Score

Has history of FA symptom onset <= 25 years of age

Is willing to agree to the following rules for use of omaveloxolone (Skyclarys): 1. For a candidate who is currently taking omaveloxolone, has been on a stable dose for 12 weeks, expects to continue taking omaveloxolone at that dose throughout the study, and is willing to stop taking omaveloxolone at the direction of the Investigator or Sponsor's Medical Monitor if evidence of transaminitis or synthetic liver dysfunction is detected during the study 2. For a candidate who is not actively taking omaveloxolone, at least 12 weeks have passed since the last dose and the candidate agrees not to resume omaveloxolone during the 18-month period after SGT-212 infusion NOTE: The use of any other approved or investigational medicinal product for the treatment of FA should be discussed with the study team

12 exclusion criteria prevent from participating

Antibodies against adeno-associated virus serotype 9 (AAV9)

Has a body weight <= 25 kilogram (kg) or has body mass index (BMI) >= 33 kg/m^2

Has a contraindication to endomyocardial biopsy (EMB) or cardiac catheterization

Has a modified FARS (mFARS) score < 20

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

3 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Non-ambulatory participants will receive bilateral intradentate infusion (IDN) followed by systemic intravenous (IV) infusion.

Group II

Experimental

Ambulatory participants will receive bilateral IDN infusion followed by systemic IV infusion.

Group III

Experimental

Participants will receive bilateral IDN followed by systemic IV infusion.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 3 locations

Recruiting

The Ohio State University

Columbus, United StatesOpen The Ohio State University in Google Maps

Recruiting

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, United States

Recruiting soon

The University of California, Los Angeles (UCLA)

Los Angeles, United States

Recruiting

3 Study Centers